By Maria Iglesias (Analyst, Health Advances GmbH)
Early Access Programs (EAPs) grant patients and physicians immediate use of medical products that are seeking marketing authorization (MA) or undergoing clinical trials in the European Union or elsewhere. These programs are intended for patients who have exhausted all alternative treatment options and do not match clinical trial entry criteria. There are two types of EAPs in Europe:
(1) Compassionate Use Programs are initiated by pharmaceutical companies for a group of patients in a selected clinic or hospital.
(2) Named-Patient Programs are granted in response to requests by physicians on behalf of specific or “named” patients.
Overview of Existing Programs
Compassionate Use Programs were initiated in Germany in July 2010. Today, there are 8 cohort compassionate use programs, mostly in oncology. While the process for the cohort program is more complex than the named-patient program, it grants the manufacturer a higher degree of control such as limiting the medication to a particular patient subgroup.
Table 1. Overview of the Existing EAPs in Germany
The Temporary Utilization Program (ATU) has been in place since 1994 in France. In 2016, there were 25 cohort ATUs, and 205 new medicines were available through named-patient ATUs. Reimbursement opportunities make compassionate use programs in France potentially the most attractive in Europe.
Table 2. Overview of the Existing EAPs in France
The Early Access to Medicines Scheme program was launched in 2014 in the UK, and by today, a total of 51 programs have been approved. The application process for manufacturers requires submission of a dossier with the latest available data, and evidence requirements for both regulatory purposes and NICE appraisal, thus potentially contributing to the data required for MA application and subsequent appraisal.
Table 3. Overview of the Existing EAPs in the UK
Considering the differences in EAPs per country, manufacturers will need to contemplate the opportunities and implications that each represents towards an early access to market. While early stakeholder engagement might be among the largest perceived benefits today, more progress from both healthcare systems and industry could potentially increase program attractiveness by capitalizing on the opportunity for concurrent early generation of real-world evidence.
About the Author
Maria Iglesias is a member of Health Advances European Practice, which helps clients navigate through the diversity of European healthcare systems to optimize commercialization strategies of pipeline and in-market products.
- European Medicines Agency, Committee for Medical Products for Human Use, Guideline on the Compassionate Use of Medicinal Products, Pursuant to Article 83 of Regulation (EC) No 726/2004
- Federal Institute for Drugs and Medical Devices, The German Medicinal Product Act
- French National Agency for Medicines and Health Products Safety, Annual Reports
- Gov.uk, Office for Life Sciences, Early Access to Medicines Scheme Guidance